Half of newly approved drugs exempt from pediatric testing

Key takeaways:

• Out of 323 drugs, only 10% completed testing in children before receiving FDA approval.
• Rare disease designations exempt companies from pediatric testing, but that may soon change.

Less than half of drugs approved between 2015 and 2021 were required to conduct pediatric trials under the Pediatric Research Equity Act, according to findings published in JAMA.

Congress passed the Pediatric Research Equity Act (PREA) in 2003 requiring drug manufacturers to conduct pediatric trials before submitting applications to the FDA, but there are multiple ways drug manufacturers can get out of those requirements, according to the researchers.

“The law exempted drugs indicated for rare disease, allowed deferred trials for drugs otherwise ready for use in adults and permitted trial waivers for certain impractical circumstances, such as for diseases that do not affect children,” Ian T.T. Liu, MD, JD, MPH, MS, from the division of pharmacoepidemiology and pharmacoeconomics at Brigham and Women’s Hospital, and colleagues wrote. “Studies have shown that waivers and deferrals of PREA requirements are common, while postapproval pediatric testing is often delayed.”

The researchers examined the postapproval testing requirements for drugs that received FDA approval from 2015 through 2021 and tracked the progress of related pediatric trials. Median follow-up was 5.6 years (interquartile range [IQR], 3.97-7.15).

Out of 323 drugs, 237 (73%) were approved for adults only. Overall, 124 (38%) had rare disease designation and were exempt from pediatric testing, and 70 (22%) had the testing requirements waived. Thirty-two drugs (10%) had completed pediatric testing requirements before approval, whereas 97 (30%) were required to conduct postapproval pediatric testing.

At follow-up, one-quarter (28%) of pediatric trials were complete, 53% were still ongoing and 17% were released before completion.

The researchers found that nearly half (41%) of ongoing pediatric trials had surpassed their original estimated completion date, which were delayed by a median of 3 years (IQR, 1.69-3.67).

Between 1999 and 2003, before PREA, 15% of drugs had rare disease designations. The proportion rose to 23% between 2003 and 2012, and nearly half (46%) of drugs approved between 2015 and 2021 had rare disease designation, which the researchers noted may be limiting the scope of PREA.

That could change soon, though. As Healio previously reported, Congress eliminated the rare disease exemption for adult cancer drugs in 2020, which resulted in more cancer drugs being tested in pediatric populations. According to Liu and colleagues, legislators introduced the Innovation in Pediatric Drugs Act this year, which would remove the rare disease exemption for other types of drugs.

“This study suggests that elimination of the rare disease exemption may increase pediatric testing requirements for new drugs, although additional reforms may be necessary,” the researchers wrote.